Published in:
Open Access
01-11-2013 | Meeting abstract
PW02-027 - CAPS and cost-effectiveness analysis project
Authors:
O Della Casa Alberighi, L Accame, J Frenkel, M Gattorno, A Martini, B Neven, P Quartier, F Pierotti, G Turchetti
Published in:
Pediatric Rheumatology
|
Special Issue 1/2013
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Excerpt
Ultra-orphan drugs are medicines used to treat exceptionally rare diseases that are chronically debilitating or life-threatening. Low patient numbers make it difficult for pharmaceutical companies to recoup research and development costs, and consequently these medicines are generally very expensive on a per patient basis. European Union (EU) regulations promote the development of orphan drugs; but to contain costs, EU healthcare systems will increasily need the cost-effectiveness analysis (CEA) of therapies when deciding if they should be funded. Conventional methods for CEA of drugs for common conditions do not apply to ultra-orphan drugs; therefore, additional factors need to be considered. …
